Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Titre officiel

A Phase III, Multicentre, International Study With a Parallel, Randomised, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)

Sommaire:

A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.

Description de l'essai

Primary Outcome:

• Confirmed Objective Response Rate (ORR) for Arm A versus Arm B

Secondary Outcome:

• Change in chronic target PN pain intensity from baseline for Arm A versus Arm B as assessed using a PRO questionnaire
• Duration of response (DoR) for Arm A
• Progression Free Survival (PFS) for Arm A
• Time to progression (TTP) for Arm A
• Time to Response (TTR) for Arm A
• Target PN volume for Arm A vs Arm B
• Physical functioning assessed using PROMIS physical function items
• Health Related Quality of Life (HRQoL) outcomes assessed using PlexiQoL

This is a randomized, double-blind, placebo-controlled, 2 arm multicentre, global Phase III study to assess the efficacy and safety of selumetinib compared with placebo in adult participants with NF1 who have symptomatic, inoperable PN.

Voir cet essai sur ClinicalTrials.gov